Hematopoietic Stem Cell Support in Patients With Refractory Sarcoidosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00282438
• Date of registration: 24/01/2006
• Prospective Registration: No
• Primary sponsor: Northwestern University
• Public title: Hematopoietic Stem Cell Support in Patients With Refractory Sarcoidosis
• Scientific title: Hematopoietic Stem Cell Transplant in Patients With Refractory Sarcoidosis: A Phase I/II Trial
• Date of first enrolment: December 2003
• Target sample size: 2
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00282438
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Richard Burt, MD
• Affiliation: Northwestern University

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age = 18years and = 60 years at the time of pretransplant evaluation.

2. Definitive diagnosis of sarcoidosis in pathologic specimen.

3. Patients who failed to respond to conventional treatment of at least 3 months duration
with corticosteroids (equivalent dosage of prednisone 1.0mg/kg/day to start). Patients
must also have failed two or more of the followings: TNF inhibitors (etanercept,
infliximab), methotrexate, azathioprine, 6-MP, cyclosporin, tacrolimus, mycophenolate
mofetil, gold, dapsone, colchicine, chloroquine/hydroxychloroquine or any other
immunosuppressive or modulating drugs.

4. Failure of therapy defined by (not caused by unrelated conditions) any one of
following:

- Progressive pulmonary disease (stage II or III) defined by decline in pulmonary
function (DLCo, VC or FEV1) of 15% or more over 12 months.

- Progressive CNS disease (worsening symptoms such as paraparesis or medically
refractory seizure).

- Persistent peripheral neuropathy (one of following):

1. Persistent muscle weakness Grade 3/5 or worse (MRC) in at least one movement
(e.g. ankle dorsiflexion) in two limbs.

2. Persistent cranial nerve involvement such as persistent facial diplegia.

3. Persistent incapacitating sensory loss (e.g. gait ataxia, falls > 1/month).

- Progressive loss of vision.

- Persistent hypercalcemia.

5. Cardiac sarcoidosis that is proven by cardiac biopsy or cardiac MRI.

Exclusion Criteria:

1. Alternative diagnosis.

2. Noncompliance to medical care.

3. > 10 pack-year history of cigarette smoking if lung disease is the major problem.

4. Poor performance (PS) status (ECOG >2) at the time of entry, unless decline of PS is
due to the disease itself.

5. Significant end organ damage such as:

1. Overt congestive heart failure (NYHA Class III or IV).

2. Active ischemic heart disease, s/p myocardial infarction within 6 months, s/p
unstable angina within 3 months, s/p CVA within 6 months, s/p hospitalization for
CHF within 3 months.

3. Untreated life-threatening arrhythmia.

4. Pulmonary hypertension > 40 mmHg.

5. End-stage lung disease (TLC < 55%, FVC < 55%, or DLCO < 40% of predicted value).

6. Serum creatinine > 2.5 or creatinine clearance < 30 ml/min.

7. Liver cirrhosis, transaminases > 3x normal or bilirubin > 2.0 unless due to
Gilbert's disease.

6. HIV positive.

7. Uncontrolled diabetes mellitus, or any other illness that in the opinion of the
investigators would jeopardize the ability of the patient to tolerate aggressive
treatment.

8. Prior history of malignancy except localized basal cell or squamous skin cancer. Other
malignancies for which the patient is judged to be cured by local surgical therapy,
such as (but not limited to) head and neck cancer, or stage I or II breast cancer will
be considered on an individual basis.

9. Positive pregnancy test, inability or unable to pursue effective means of birth
control, failure to willingly accept or comprehend irreversible sterility as a side
effect of therapy.

10. Significant psychological issues, social issues, psychiatric illness or mental
deficiency making compliance with treatment or informed consent impossible.

11. Inability to give informed consent.

12. Major hematological abnormalities such as platelet count less than 100,000/ul, ANC
less than 1000/ul.



Age minimum: 18 Years
Age maximum: 60 Years
Gender: All

Health Condition(s) or Problem(s) studied

Sarcoidosis

Intervention(s)

Biological: Autologous hematopoietic stem cell transplantation

Biological: Allogeneic stem cell transplantation

Primary Outcome(s)

Presence of Toxicity [Time Frame: For length of hospital stay (until discharge).]

Survival [Time Frame: Participants are to be followed at 6 months and then yearly until 5 years]

Time to Disease Progression [Time Frame: Participants are to be followed at 6 months and then yearly until 5 years]

Secondary ID(s)

NU FDA SARC.2003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 24/02/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00282438